Patisiran

Summary

Patisiran (CHEMBL3989987) is a phase-3 clinical-stage oligonucleotide (ATC N07XX12); indicated across 4 conditions including amyloidosis and familial amyloid neuropathy.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• ATC class: N07XX12
• Indications: 4 conditions
• Clinical trials: 12

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3989987
Name	Patisiran
Type	Oligonucleotide
Max phase	3
ATC	N07XX12

Also known as: ALN-18328, ALN-TTR02, Genz-438027, Patisiran, SAR-438037, PATISIRAN

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

4 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
amyloidosis	3	MONDO:0019065	EFO:1001875
familial amyloid neuropathy	3	MONDO:0007100	EFO:0004129
hereditary amyloidosis	3	MONDO:0018634	MONDO:0018634
diabetic neuropathy	0	MONDO:0006626	EFO:1000783

Clinical trials

Total trials: 12.

Phase distribution

Phase	Trials
PHASE3	5
Not specified	3
PHASE1	2
PHASE2	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01960348	PHASE3	COMPLETED	APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
NCT02510261	PHASE3	COMPLETED	The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
NCT03759379	PHASE3	COMPLETED	HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
NCT03862807	PHASE3	COMPLETED	Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
NCT03997383	PHASE3	COMPLETED	APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
NCT01617967	PHASE2	COMPLETED	Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
NCT01559077	PHASE1	COMPLETED	Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects
NCT02053454	PHASE1	COMPLETED	A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
NCT05023889	EARLY_PHASE1	COMPLETED	Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
NCT02939820	Not specified	APPROVED_FOR_MARKETING	Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
NCT04201418	Not specified	COMPLETED	A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
NCT05505838	Not specified	NO_LONGER_AVAILABLE	Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).